Contributions
Abstract: EP1053
Type: E-Poster Presentation
Session title: Myeloma and other monoclonal gammopathies - Clinical
Background
Immunoglobulin light chain (AL) amyloidosis is a heterogeneous disease. The prognosis is highly related to the stage of the disease which, in turn, is dependent on the hearth involvement. At diagnosis, patients in Mayo stage III and IV represent the majority of AL amyloidosis, and are defined 'high-risk' because their outcome is poor.
Aims
From 2003 to 2019, 111 patients were diagnosed with systemic AL amyloidosis in our Center; 52 patients were identified as 'high risk'. We retrospectively analyzed the impact of hematological responses (HeR) by day 30 on overall survival (OS). All patients underwent first line bortezomib-based chemotherapy (6 cycles, weekly administration).
Methods
Baseline characteristics are shown in Table 1. All patients had an initial dFLC >50 mg/L, and a NTproBNP value above the normal limit. Diagnosis of amyloidosis was biopsy-proven and amyloid typing was established by immunoelectron microscopy in all cases. Patients were considered to have cardiac amyloidosis based on the accepted criteria for the definition of organ involvement. Hematologic and organ responses (OR) were defined according to Palladini et al.
Results
By day 30 of treatment, 23 patients (44%) achieved a HeR (11 CR and 12 VGPR). Twenty-nine patients (56%) attained PR or NR (14 and 15 patients, respectively). At 6 months 21 out of 29 patients with an inadequate response at day 30 did not change their hematological status (7 and 14 of them were PR and NR respectively at day 30), and 8 out of these 21 patients experienced early (i.e., within 6 mo.) death. All these latter patients were NR at day 30. The remaining 8 patients (7 PR and 1 NR at day 30) achieved a VGPR with no treatment modification. Remarkably, 12 out of 14 PR patients at day 30 achieved an organ response at the end of treatment; 7 of them achieved a VGPR while in 5 the hematological status did not change. The median OS of patients achieving the HeR, PR and NR at day 30 were 43, 24, 11 months, respectively.
At univariate analysis, achieving a HeR and PR at day 30 significantly predicted survival, with respect to NR (respectively p=0.001 and p=0.007). These results were confirmed even in the final multivariate Cox model (HeR vs NR p=0.001 and PR vs NR p=0.04).
Conclusion
In this limited retrospective analysis, HeR (CR+VGPR) at day 30 confirmed its prognostic value. Nevertheless, patients who reached a suboptimal response (PR) at day 30 have reasonable chances of survival, and do not necessarily need a change in therapeutic strategy even in the face of permissive clinical conditions. Therefore we confirm that achieving a rapid HeR and, to a lesser extent, a PR to bortezomib at day 30 in patients in newly diagnosed high-risk AL amyloidosis, has a favorable prognostic value. On the other hand, an early identification of NR patients should prompt a shift in therapy trying to improve the well-known dismal prognosis of these patients.
Keyword(s): AL amyloidosis
Abstract: EP1053
Type: E-Poster Presentation
Session title: Myeloma and other monoclonal gammopathies - Clinical
Background
Immunoglobulin light chain (AL) amyloidosis is a heterogeneous disease. The prognosis is highly related to the stage of the disease which, in turn, is dependent on the hearth involvement. At diagnosis, patients in Mayo stage III and IV represent the majority of AL amyloidosis, and are defined 'high-risk' because their outcome is poor.
Aims
From 2003 to 2019, 111 patients were diagnosed with systemic AL amyloidosis in our Center; 52 patients were identified as 'high risk'. We retrospectively analyzed the impact of hematological responses (HeR) by day 30 on overall survival (OS). All patients underwent first line bortezomib-based chemotherapy (6 cycles, weekly administration).
Methods
Baseline characteristics are shown in Table 1. All patients had an initial dFLC >50 mg/L, and a NTproBNP value above the normal limit. Diagnosis of amyloidosis was biopsy-proven and amyloid typing was established by immunoelectron microscopy in all cases. Patients were considered to have cardiac amyloidosis based on the accepted criteria for the definition of organ involvement. Hematologic and organ responses (OR) were defined according to Palladini et al.
Results
By day 30 of treatment, 23 patients (44%) achieved a HeR (11 CR and 12 VGPR). Twenty-nine patients (56%) attained PR or NR (14 and 15 patients, respectively). At 6 months 21 out of 29 patients with an inadequate response at day 30 did not change their hematological status (7 and 14 of them were PR and NR respectively at day 30), and 8 out of these 21 patients experienced early (i.e., within 6 mo.) death. All these latter patients were NR at day 30. The remaining 8 patients (7 PR and 1 NR at day 30) achieved a VGPR with no treatment modification. Remarkably, 12 out of 14 PR patients at day 30 achieved an organ response at the end of treatment; 7 of them achieved a VGPR while in 5 the hematological status did not change. The median OS of patients achieving the HeR, PR and NR at day 30 were 43, 24, 11 months, respectively.
At univariate analysis, achieving a HeR and PR at day 30 significantly predicted survival, with respect to NR (respectively p=0.001 and p=0.007). These results were confirmed even in the final multivariate Cox model (HeR vs NR p=0.001 and PR vs NR p=0.04).
Conclusion
In this limited retrospective analysis, HeR (CR+VGPR) at day 30 confirmed its prognostic value. Nevertheless, patients who reached a suboptimal response (PR) at day 30 have reasonable chances of survival, and do not necessarily need a change in therapeutic strategy even in the face of permissive clinical conditions. Therefore we confirm that achieving a rapid HeR and, to a lesser extent, a PR to bortezomib at day 30 in patients in newly diagnosed high-risk AL amyloidosis, has a favorable prognostic value. On the other hand, an early identification of NR patients should prompt a shift in therapy trying to improve the well-known dismal prognosis of these patients.
Keyword(s): AL amyloidosis